Safety of Risdiplam in Japanese Patients with Spinal Muscular Atrophy: A 12‑Month Interim Analysis of a Postmarketing Surveillance Study
This 12-month interim analysis of PMS data indicated that risdiplam was well tolerated among Japanese patients with SMA, consistent with previous clinical trial findings. A comprehensive evaluation of the safety and efficacy of risdiplam will be provided in the final 24-month analysis.
Authors
- Hisahide Nishio (ORCID: https://orcid.org/0000-0002-6778-4511)
- Kayoko Saito (ORCID: https://orcid.org/0000-0003-2632-0873)
- Masahisa Katsuno (ORCID: https://orcid.org/0000-0001-9453-9311)
- Yuki Miyano (ORCID: https://orcid.org/0000-0002-9964-6690)
- Reiko Arakawa (ORCID: https://orcid.org/0000-0002-0910-8910)
- Toshio Saito (ORCID: https://orcid.org/0000-0002-0432-0402)
- Hirofumi Komaki (ORCID: https://orcid.org/0000-0002-0659-1417)
- Yasuhiro Takeshima (ORCID: https://orcid.org/0000-0002-6468-7185)
- Yuka Ishikawa (ORCID: https://orcid.org/0000-0002-1406-9768)
- Takahiko Tsumuraya (ORCID: https://orcid.org/0009-0009-7261-0346)
- Hiroki Kawata
Institutions
- Hyogo Medical University (JP)
- Kobe Gakuin University (JP)
- National Center for Global Health and Medicine (JP)
- National Hospital Organization Hokkaido Medical Center (JP)
- National Center of Neurology and Psychiatry (JP)
- Toneyama National Hospital (JP)
- Chugai Pharma (United States) (US)
- Tokyo Women's Medical University (JP)
- Hyogo Prefectural Nishinomiya Hospital (JP)
- Nagoya University (JP)
- Hyogo University (JP)
Publication Details
- Journal
- Neurology and Therapy
- Published
- 2025-08-09
- DOI
- https://doi.org/10.1007/s40120-025-00795-x
- Primary Topic
- Neurogenetic and Muscular Disorders Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Chugai Pharmaceutical